Pilot Study Evaluating Safety & Efficacy of DCBT: NiCord® & UNM CBU to Patients With Hematological Malignancies

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Gamida Cell ltd
ClinicalTrials.gov Identifier:
NCT01221857
First received: October 14, 2010
Last updated: February 24, 2014
Last verified: February 2014
  Purpose

Pilot Study Evaluating the Safety and Efficacy of a Co-Transplantation of NiCord®, a UCB-derived ex Vivo Expanded Population of Stem and Progenitor Cells with a Second, Unmanipulated CBU in Patients with Hematological Malignancies


Condition Intervention Phase
Acute Lymphoblastic Leukemia (ALL)
Acute Myelogenous Leukemia (AML)
Myelodysplastic Syndrome (MDS)
Non-Hodgkin's Lymphoma
Hodgkin's Disease
Drug: NiCord®
Phase 1
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Combination With a Second, Unmanipulated Cord Blood Unit in Patients With Hematological Malignancies

Resource links provided by NLM:


Further study details as provided by Gamida Cell ltd:

Primary Outcome Measures:
  • Safety and Tolerability: will be measured by acute NiCord® infusional toxicity, and assessment of the proportion of patients with neutrophil engraftment [ Time Frame: 180 days post-transplant ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Proportion of non-relapse mortality, development of acute GvHD [ Time Frame: 100 days post-transplant ] [ Designated as safety issue: Yes ]

Enrollment: 11
Study Start Date: November 2010
Study Completion Date: May 2013
Primary Completion Date: September 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NiCord Drug: NiCord®
NiCord® is a cell-based product composed of umbilical cord-derived ex vivo expanded stem and progenitor cells.

Detailed Description:

Allogeneic hematopoietic stem cell transplantation (HSCT) is a potentially curative procedure for various hematological malignancies, bone marrow failure syndromes and inherited metabolic disorders. The application of allogeneic HSCT is limited by donor availability such that only approximately one-third of the otherwise appropriate candidates have suitably matched family donors. Alternative donors include mismatched family members or matched unrelated donors, but these approaches are often complicated by an increased risk of graft-versus-host disease (GvHD) and a prolonged and cumbersome search and procurement process. In addition, far fewer subjects of racial minorities find suitable human leukocyte antigen (HLA)-matched donors.

Umbilical cord blood has been increasingly used as an alternative source of stem cells and has extended the availability of allogeneic HSCT to patients who would otherwise not be eligible for this curative approach. In the last decade the number of cord blood transplantations from related and unrelated donors has increased dramatically. It is estimated that more than 20,000 patients have undergone cord blood transplantation from unrelated donors to date for a variety of genetic, hematological, immunological, metabolic and oncologic disorders. The major advantages of cord blood transplantation include easy procurement, no risk to donors, reduced incidence of transmitting infections, immediate availability, and reduced risk of acute GvHD in the setting of donor-recipient HLA mismatch. Nevertheless, the low cell dose remains a main limitation of this cell source leading to delayed hematopoietic reconstitution, higher risk of graft failure and relatively high treatment related mortality rates as compared to other hematopoeitic cell sources. To improve outcomes and extend applicability of cord blood transplantation, one potential solution is ex vivo expansion of cord blood-derived stem and progenitor cells.

The Sponsor has undertaken to develop NiCord®, which is based on a novel technology for ex vivo cell expansion of cord blood derived hematopoietic progenitor cells. By increasing the number of the short and long-term reconstitution progenitor cells transplanted, NiCord® has the potential to enable broader application of umbilical cord blood transplantation and improve clinical outcomes in subjects with high-risk hematological malignancies.

The main objective of the current study is to evaluate the safety of co-transplantation of NiCord® and an unmanipulated cord blood unit in patients with hematological malignancies following myeloablative therapy.

  Eligibility

Ages Eligible for Study:   8 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Applicable disease and eligible for myeloablative SCT
  • Patients must have two partially HLA-matched CBUs
  • Back-up stem cell source
  • Adequate Karnofsky Performance score or Lansky Play-Performance scale
  • Sufficient physiological reserves
  • Signed written informed consent

Exclusion Criteria:

  • HLA-matched related donor able to donate
  • Prior allogeneic HSCT
  • Lymphoma patients with progressive disease
  • Other active malignancy
  • Human immunodeficiency virus (HIV) infection
  • Active or uncontrolled infection
  • Active/symptoms of central nervous system (CNS) disease
  • Pregnancy or lactation
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01221857

Locations
United States, Illinois
Loyola University, Cardinal Bernardin Cancer Center
Maywood, Illinois, United States, 60153
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27705
Sponsors and Collaborators
Gamida Cell ltd
Investigators
Study Director: David Snyder, PhD Gamida Cell ltd
Principal Investigator: Joanne Kurtzberg, MD Duke University
Principal Investigator: Mitchell Horwitz, MD Duke University
Principal Investigator: Patrick Stiff, MD Loyola University
  More Information

Additional Information:
No publications provided by Gamida Cell ltd

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Gamida Cell ltd
ClinicalTrials.gov Identifier: NCT01221857     History of Changes
Other Study ID Numbers: GC P#01.01.020
Study First Received: October 14, 2010
Last Updated: February 24, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Gamida Cell ltd:
Double Umbilical Cord Blood Stem Cell Transplantation
Hematological Malignancies
HLA Mismatched Donors
Cord Blood Transplantation

Additional relevant MeSH terms:
Hodgkin Disease
Leukemia
Leukemia, Lymphoid
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Lymphoma, Non-Hodgkin
Myelodysplastic Syndromes
Neoplasms
Precursor Cell Lymphoblastic Leukemia-Lymphoma
Preleukemia
Bone Marrow Diseases
Hematologic Diseases
Immune System Diseases
Immunoproliferative Disorders
Lymphatic Diseases
Lymphoma
Lymphoproliferative Disorders
Neoplasms by Histologic Type
Precancerous Conditions

ClinicalTrials.gov processed this record on October 23, 2014